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Drug Category: Endocrine and Metabolic Agents

Medication Class/Individual Agents: Enzyme Disorder and Substrate Reduction Therapies

I. Prior-Authorization Requirements

 Enzyme Disorder Therapies – Injectable Agents

Clinical Notes

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

agalsidase beta Fabrazyme PA  
alglucosidase alfa Lumizyme PA  
asfotase alfa Strensiq PA  
elapegademase-lvlr Revcovi PA  
elosulfase alfa Vimizim PA  
galsulfase Naglazyme PA  
idursulfase Elaprase PA  
imiglucerase Cerezyme PA  
laronidase Aldurazyme PA  
pegademase bovine Adagen PA  
pegvaliase-pqpz Palynziq PA  
taliglucerase alfa Elelyso PA  
velaglucerase alfa Vpriv PA  
vestronidase alfa-vjbk Mepsevii PA  

Please note: In the case where the prior authorization (PA) status column indicates PA, both the brand and generic (if available) require PA. Typically, the generic is preferred when available unless the brand-name drug appears on the MassHealth Brand Name Preferred Over Generic Drug List. In general, when requesting the non-preferred version, whether the brand or generic, the prescriber must provide medical records documenting an inadequate response or adverse reaction to the preferred version, in addition to satisfying the criteria for the drug itself.

  • Lysosomal storage disorders are caused by a deficiency or absence of required enzymes. The consequence is an accumulation of compounds that are normally degraded, causing cell and organ dysfunction. Before the development of enzyme replacement therapy, management of these conditions consisted of supportive care and treatment of the complications.
  • A number of exogenously supplied enzymes are available for lysosomal storage disorders including adenosine deaminase (ADA) deficiency, Gaucher disease, Fabry disease, Hunter syndrome, hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthetase, hypophosphatasia, lysosomal acid lipase deficiency, mucopolysaccharidosis type I, IVA, VI, and VII, and Pompe disease.
  • Pancreatic enzyme replacement is indicated for the treatment of exocrine pancreatic insufficiency due to cystic fibrosis or other conditions. Multiple formulations of pancreatic enzymes exist with different combinations of lipase, protease, and amylase; however, these enzymes may differ in their effects. Patients should be reevaluated after any changes in enzyme preparation or dose.
 

 Enzyme Disorder Therapies – Oral Agents

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

carglumic acid Carbaglu PA  
glycerol phenylbutyrate Ravicti PA  
migalastat Galafold PA  
pancrelipase-Creon DR Creon DR test  
pancrelipase-Pancreaze DR Pancreaze DR test  
pancrelipase-Pertzye DR Pertzye DR test  
pancrelipase-Viokace Viokace test  
pancrelipase-Zenpep DR Zenpep DR test  
sapropterin Kuvan PA  

 Substrate Reduction Therapies

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

eliglustat Cerdelga PA  
miglustat Zavesca BP PA  
sebelipase alfa Kanuma PA  
Table Footnotes
BP Brand Preferred over generic equivalents. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing the non-preferred drug generic equivalent.
 

II. Therapeutic Uses

FDA-approved, for example: 

  • Adenosine deaminase severe combined immunodeficiency (ADA-SCID) (Adagen, Revcovi)
  • Fabry disease (Fabrazyme, Galafold)
  • Gaucher Disease Type 1 (Cerdelga, Cerezyme, Elelyso, miglustat, Vpriv)
  • Hunter Syndrome (Elaprase)
  • Hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthetase (NAGS) (Carbaglu)
  • Hypophosphatasia (Strensiq)
  • Lysosomal acid lipase deficiency (Kanuma)
  • Mucopolysaccharidosis I (Aldurazyme)
  • Mucopolysaccharidosis IVA (Morquio A syndrome) (Vimizim)
  • Mucopolysaccharidosis VI (Naglazyme)
  • Mucopolysaccharidosis VII (Sly syndrome) (Mepsevii)
  • Phenylketonuria (Kuvan, Palynziq)
  • Pompe disease (Lumizyme)
  • Urea cycle disorder (Ravicti)

Note: The above list may not include all FDA-approved indications.

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III.  Evaluation Criteria for Approval

Please note: In the case where the prior authorization (PA) status column indicates PA, both the brand and generic (if available) require PA. Typically, the generic is preferred when available unless the brand-name drug appears on the MassHealth Brand Name Preferred Over Generic Drug List. In general, when requesting the non-preferred version, whether the brand or generic, the prescriber must provide medical records documenting an inadequate response or adverse reaction to the preferred version, in addition to satisfying the criteria for the drug itself.

  • All PA requests must include clinical diagnosis, drug name, dose, and frequency.
  • A preferred drug may be designated for this therapeutic class. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing a non-preferred drug within a therapeutic class. Additional information about these agents, including PA requirements and preferred products, can be found within the MassHealth Drug List at www.mass.gov/druglist.
  • For recertification requests, approval may require submission of additional documentation including, but not limited to, documentation of: some or all criteria for the original approval; response to therapy; clinical rationale for continuation of use; status of member’s condition; appropriate diagnosis; appropriate age; appropriate dose, frequency, and duration of use for requested medication; complete treatment plan; current laboratory values; and member’s current weight.
  • Additional criteria may apply, depending upon the member’s condition and requested medication (see below).

      

Adagen

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • laboratory results documenting one of the following:
      • absent ADA enzymatic activity in lysed erythrocytes; or 
      • elevated levels of adenosine and deoxyadenosine in the urine and plasma; or 
      • a marked increase in deoxyadenosine triphosphate (dATP) levels in erythrocyte lysates; or 
      • a significant decrease in ATP concentration in red blood cells; or 
      • absent or extremely low levels of N adenosylhomocysteine hydrolase in red blood cells; or 
      • severe T cell deficiency manifested by lymphopenia and poor T cell responses to mitogens and antigens; or 
      • absent thymic shadow on chest radiograph; and
    • member's current weight.

Aldurazyme

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • results from an enzyme assay test showing reduced lysosomal alpha-L-iduronidase activity in peripheral blood leukocytes, plasma, or cultured fibroblasts; and
    • member's current weight.

Carbaglu

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • appropriate dosing; and
    • results from genetic test or an enzyme assay test supporting the diagnosis.

Cerdelga

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • results from enzyme assay test showing reduced activity of glucocerebrosidase; and
    • member is not currently receiving enzyme replacement therapy.

Cerezyme and Vpriv

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • results from an enzyme assay test showing reduced activity of the enzyme glucocerebrosidase; and
    • member's current weight.

Elaprase

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • results from an iduronate-2-sulfatase assay test showing reduced or absent activity in the serum, white blood cells, or fibroblasts; and
    • member's current weight.

Elelyso

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • results from an enzyme assay test showing reduced activity of the enzyme glucocerebrosidase; and
    • member is ≥ 18 years of age; and
    • member's current weight.

Fabrazyme

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • results from an enzyme assay test showing reduced or absent α-galactosidase A (α-GAL) enzyme activity in plasma, leukocytes, tears, or biopsied tissue; and
    • member's current weight.

Galafold

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • prescriber is a specialist (e.g., clinical genetics specialist or nephrologist) or consult is provided; and
    • results from an enzyme assay test showing reduced or absent α-galactosidase A (α-GAL) enzyme activity in plasma, leukocytes, tears, or biopsied tissue; and
    • member has GLA variants which are amenable to treatment with the requested agent; and
    • request is within quantity limit of 15 units/30 days.

Kanuma

  • Documentation of all of the following is required:
    • diagnosis of lysosomal acid lipase deficiency; and
    • member's current weight.

Kuvan

  • Documentation of all of the following is required:
    • appropriate diagnosisand
    • results from genetic testing or molecular analysis to confirm diagnosis; and
    • documentation that medication will be used in conjunction with a phenylalanine-restricted diet; and
    • member's current weight.

Lumizyme

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • results from acid maltase enzyme alpha-glucosidase (GAA) assay test showing reduced or absent activity from cultured skin fibroblasts, muscle biopsy, or lymphocyte testing; and
    • member's current weight.

Mepsevii

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • results from genetic testing showing mutations in the beta glucuronidase gene; and
    • member's current weight.

miglustat

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • results from enzyme assay test showing reduced activity of glucocerebrosidase; and
    • contraindication to enzyme replacement therapy.

Naglazyme

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • results from an enzyme assay test showing reduced arylsulfatase B (ASB) enzyme activity in leukocytes or fibroblasts along with elevated urine glycosaminoglycan (GAG) levels; and
    • member's current weight.

Palynziq

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • results from genetic testing or molecular analysis to confirm diagnosis; and
    • blood phenylalanine concentrations > 600 micromol/L; and
    • medication will be used in conjunction with a phenylalanine-restricted diet; and
    • inadequate response, adverse reaction, or contraindication to sapropterin.

Ravicti

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • results from genetic test or an enzyme assay test supporting the diagnosis; and
    • appropriate dosing; and
    • inadequate response, adverse reaction, or contraindication to sodium phenylbutyrate.

Revcovi

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • laboratory results documenting one of the following:
      • absent ADA enzymatic activity in lysed erythrocytes; or 
      • elevated levels of adenosine and deoxyadenosine in the urine and plasma; or 
      • a marked increase in deoxyadenosine triphosphate (dATP) levels in erythrocyte lysates; or 
      • a significant decrease in ATP concentration in red blood cells; or 
      • absent or extremely low levels of N adenosylhomocysteine hydrolase in red blood cells; or 
      • severe T cell deficiency manifested by lymphopenia and poor T cell responses to mitogens and antigens; or 
      • absent thymic shadow on chest radiograph; and
    • member's current weight; and
    • one of the following:
      • inadequate response, adverse reaction, or contraindication to Adagen; or
      • clinical rationale for use of the requested agent instead of Adagen.

Strensiq

  • Documentation of all of the following is required:
    • diagnosis of perinatal-onset, infantile-onset, or juvenile-onset hypophosphatasia; and
    • member's current weight.

Vimizim

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • member is ≥ five years of age; and
    • results from an enzyme assay test showing reduced N-acetylgalactosamine-6-sulfatase activity in blood and/or skin cells; and
    • member's current weight.


Original Effective Date: 11/2012

Last Revised Date: 11/2019


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Last updated 11/25/19